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Early Intervention and Speed-to-Effect in Spinal Muscular Atrophy Type 1 Following Onasemnogene Abeparvovec Gene Replacement Therapy: Results of aPost-Hoc Analysis of Pooled Clinical Study Data

Crossref DOI link: https://doi.org/10.1007/s40120-025-00791-1

Published Online: 2025-07-07

Published Print: 2025-10

Update policy: https://doi.org/10.1007/springer_crossmark_policy

Authors

Toro, Walter

Reyna, Sandra P.

Ritter, Shannon

Patel, Anish

Mumneh, Nayla

Dabbous, Omar
Funding

Funding for this research was provided by:

Novartis Pharma AG
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Text and Data Mining valid from 2025-07-07

Version of Record valid from 2025-07-07
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Article History

Received: 14 May 2025

Accepted: 17 June 2025

First Online: 7 July 2025

Declarations

:

: Walter Toro, Sandra P. Reyna, and Nayla Mumneh are employees of Novartis and own stock/other equities. Shannon Ritter, Anish Patel, and Omar Dabbous were employees of Novartis at the time of this study and own stock/other equities. Anish Patel is currently an employee of Nanoscope Therapeutics Inc. Omar Dabbous is currently an employee of Komar Pharmaceuticals and Curestack.

: The START [], STR1VE-US [], and STR1VE-EU [] study protocols were approved by the institutional review boards at each participating institution (Table ), and study procedures were conducted according to the principles outlined in the Declaration of Helsinki. The RESTORE registry is being conducted in accordance with established research principles, local treatment practices and regulations, and guidelines of the International Council on Harmonisation. All parents or guardians provided written informed consent before any study procedures were performed.

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